Tolerx and GSK Announce Results from Phase III DEFEND-1 Trial

Tolerx and GSK Announce Results from Phase III DEFEND-1 Trial

March 11, 2011

NEW YORK, March 11, 2011- JDRF’s industry partner Tolerx, Inc., a Cambridge, MA-based biopharmaceutical company, and its commercial partner GlaxoSmithKline (GSK), announced that its DEFEND-1 Phase III clinical trial of the anti-CD3 drug, otelixizumab, did not meet its primary efficacy endpoint of change in C-peptide levels at 12 months in recently diagnosed patients with type 1 diabetes. Measurement of C-peptide proteins show how much insulin the body is producing.

The goal of the randomized, placebo-controlled DEFEND-1 trial was to evaluate the safety and efficacy of otelixizumab in preserving beta cell function and maintaining insulin production in these individuals.

Tolerx and GSK are currently reviewing the data from the DEFEND-1 trial to better understand the outcome and determine next steps. According to the companies, no new or unexpected treatment-related safety concerns emerged during the DEFEND-1 study. GSK will continue to explore additional dosing regimens to inform decisions about the future clinical development program for otelixizumab.

Tolerx is suspending recruitment and dosing of its DEFEND-2 trial, a confirmatory Phase III study of otelixizumab similar in trial design to DEFEND-1, pending review of the DEFEND-1 results.

JDRF will continue to report on ongoing developments as information becomes available.

DEFEND-1 is a randomized, placebo-controlled Phase III study of 272 patients, age 12 to 45, with new-onset type 1 diabetes. DEFEND-1 was conducted at over 100 study centers throughout North America and Europe. The study was designed to evaluate whether a single 8-day intravenous course of otelixizumab (3.1mg), administered not more than 90 days after the initial diagnosis of autoimmune type 1 diabetes, preserved the function of insulin-producing beta cells in the pancreas, as measured by C-peptide. Measurement of C-peptide (a protein that shows how much insulin the body is producing) at 12 months after dosing was the primary endpoint in DEFEND-1 and is a well established surrogate measure of beta cell function and a recommended endpoint by the U.S. Food and Drug Administration (FDA) and the American Diabetes Association.

About JDRF

JDRF is the leading global organization focused on type 1 diabetes (T1D) research. Driven by passionate, grassroots volunteers connected to children, adolescents, and adults with this disease, JDRF is now the largest charitable supporter of T1D research. The goal of JDRF research is to improve the lives of all people affected by T1D by accelerating progress on the most promising opportunities for curing, better treating, and preventing T1D. JDRF collaborates with a wide spectrum of partners who share this goal.

Since its founding in 1970, JDRF has awarded more than $1.7 billion to diabetes research. Past JDRF efforts have helped to significantly advance the care of people with this disease, and have expanded the critical scientific understanding of T1D. JDRF will not rest until T1D is fully conquered. More than 80 percent of JDRF’s expenditures directly support research and research-related education.